1. Harrison CN et al. Proc ASH 2015;Abstract 59.
Long-Term Efficacy and Safety in COMFORT-II, a Phase 3 Study Comparing Ruxolitinib with Best Available Therapy for the Treatment of Myelofibrosis: 5-Year Final Study Results
Harrison CN et al. Proc ASH 2015;Abstract 59.

2. COMFORT-II Trial: Five-Year Follow-Up of Ruxolitinib (RUX) versus Best Available Therapy (BAT) in Myelofibrosis (MF)
Five-year follow-up of a multicenter, open-label, randomized (2:1) Phase III study of RUX, a JAK inhibitor, versus BAT, with crossover (CO)
N = 219 patients with intermediate 2- or high-risk MF:
N = 146 received RUX; N = 73 received BAT (45 crossed over)
Study objectives: Five-year efficacy, safety
RUX
BAT
BAT  RUX CO
5 y on study Tx
≥35% SVR
53% (78/146)
Not reported
42%
(19/45)
67%
(34/51)
SVR = spleen volume reduction
Harrison CN et al. Proc ASH 2015;Abstract 59.

3. COMFORT-II 5-Year Follow-Up: Conclusions
Median overall survival (OS) with RUX versus BAT: Not reached versus 4.1 years (HR = 0.67; p = 0.06)
Adjusting for CO to RUX: Median OS with BAT 2.7 years (HR = 0.44)
Bone-marrow fibrosis and JAK2 burden were improved with RUX compared to BAT.
Longer-term safety was consistent with the 3-year report:
Reason for discontinuation, N (%)
RUX (n = 146)
BAT
(n = 73)
RUX after CO
(n = 45)
All combined
107 (73)
28 (38)
34 (76)
Adverse events (mainly low platelet counts, anemia)
35 (24)
5 (7)
10 (22)
Disease progression
32 (22)
4 (6)
7 (16)
Consent withdrawn
10 (7)
9 (12)
Other (including stem cell transplant)
16 (11)
6 (13)
Five-year follow-up confirms the durability of RUX benefits.
RUX treatment is being evaluated for early MF in the Phase III ReTHINK trial.
Harrison CN et al. Proc ASH 2015;Abstract 59.